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Clinical Trial News

Clinical Trial Retention Crisis: 30% Dropout Rate Costs Industry Billions and Delays Drug Development

  • Global clinical trials face a critical challenge with 30% average dropout rates, while less than 5% of eligible participants initially enroll, significantly impacting drug development timelines and costs.
  • Clinical trial complexity has increased over the past decade, with more frequent visits and data collection requirements, while rare disease focus has narrowed the eligible patient pool.
  • Removing logistical barriers and providing comprehensive patient support services could significantly improve retention rates, according to Greenphire CEO Jim Murphy.

Doxycycline Prophylaxis Shows Promise in Preventing Bacterial STIs Among High-Risk Populations

• Doxycycline prophylaxis demonstrates potential in preventing bacterial sexually transmitted infections (STIs), particularly among gay, bisexual, and other men who have sex with men. • Studies indicate that doxycycline prophylaxis can significantly reduce the incidence of syphilis and chlamydia, but further research is needed to confirm efficacy. • Ongoing studies are exploring optimal doxycycline dosage, regimen, and formulation, as well as long-term safety and the potential for antimicrobial resistance. • Experts emphasize the need for more robust data on community acceptability, behavioral risk compensation, and cost-effectiveness before broad recommendations can be made.

Minimal Residual Disease Status Validated as Surrogate Endpoint for Progression-Free Survival in Newly Diagnosed Multiple Myeloma

  • Meta-analysis of six randomized studies confirms minimal residual disease (MRD) status as a surrogate endpoint for progression-free survival (PFS) in newly diagnosed multiple myeloma (NDMM).
  • The treatment effect on MRD-negative response strongly correlated with the hazard ratio for PFS, demonstrating a high coefficient of determination of 0.97.
  • Findings support the use of MRD status to accelerate drug development by providing a faster prediction of survival outcomes in NDMM patients.
  • The study included 3283 patients and 2208 MRD samples, reinforcing the robustness of MRD as a surrogate marker for PFS.

RNA-Targeted Drug Discovery Summit to Explore Breakthrough Small Molecule Therapeutics

  • Leading pharmaceutical companies and academic institutions unite at the 2nd RNA-Targeted Drug Discovery Summit to advance novel RNA-targeted small molecule therapeutics development.
  • Industry pioneers from Pfizer, Novartis, Bristol-Myers Squibb, and other major players will address critical challenges in RNA targeting, including binding specificity and drug-like properties.
  • The summit aims to transform previously "undruggable" RNA targets into viable therapeutic opportunities through innovative approaches and RNA-protein interaction studies.

Eptinezumab Shows Promise in Preventing Chronic Migraine

A phase 2b clinical trial demonstrates that eptinezumab, an intravenous anti-calcitonin gene-related peptide monoclonal antibody, is effective and well-tolerated for the preventive treatment of chronic migraine, paving the way for phase 3 trials.

FDA Approves Pexidartinib as First Treatment for Tenosynovial Giant Cell Tumour

  • Daiichi Sankyo's pexidartinib (Turalio) becomes the first FDA-approved therapy for tenosynovial giant cell tumour (TGCT), offering a new treatment option beyond surgery.
  • Phase III ENLIVEN trial demonstrated a 38% overall response rate with pexidartinib, including 15% complete responses and 23% partial responses, compared to zero response in placebo group.
  • The approval includes a boxed warning for potential liver toxicity, requiring careful monitoring through a Risk Evaluation and Mitigation Strategy (REMS) program.

Study to Evaluate Discontinuation and Re-Treatment in Participants With Tenosynovial Giant Cell Tumor (TGCT) Previously Treated With Pexidartinib

NCT04526704CompletedPhase 4
Daiichi Sankyo
Posted 10/20/2020

Phase 3 Study of Pexidartinib for Pigmented Villonodular Synovitis (PVNS) or Giant Cell Tumor of the Tendon Sheath (GCT-TS)

NCT02371369CompletedPhase 3
Daiichi Sankyo
Posted 5/11/2015

Glembatumumab Vedotin Fails to Meet Efficacy Endpoint in Recurrent Osteosarcoma Phase II Trial

  • Glembatumumab vedotin (GV) was evaluated in a Phase II trial for patients aged 12-50 with relapsed or refractory osteosarcoma.
  • The primary endpoint of disease control at 4 months was not met, with only one partial response and two cases of stable disease observed.
  • The antibody-drug conjugate was generally well-tolerated, with rash being the most common grade III adverse event, and toxicities similar to previous studies.
  • No correlation was found between glycoprotein non-metastatic B (gpNMB) expression and response to glembatumumab vedotin in this patient population.

Glembatumumab Vedotin in Treating Patients With Recurrent or Refractory Osteosarcoma

NCT02487979CompletedPhase 2
National Cancer Institute (NCI)
Posted 2/16/2016

Genentech Expands Drug Discovery Portfolio with Three Strategic Platform Partnerships

  • Genentech has secured partnerships with Convelo Therapeutics, Sosei Heptares, and Skyhawk Therapeutics to enhance its small-molecule drug discovery capabilities across multiple therapeutic areas.
  • The Sosei Heptares collaboration, valued at up to $1 billion, focuses on GPCR drug targets, while Convelo's partnership aims to develop remyelination therapies for multiple sclerosis.
  • Skyhawk Therapeutics partnership will explore RNA-splicing modulation for cancer and neurodegenerative diseases, expanding Genentech's reach into novel therapeutic approaches.

FDA Approves Merck's Recarbrio for Complex Infections with Antimicrobial Stewardship Focus

  • The FDA has granted approval for Recarbrio, Merck's novel triple-drug antibiotic combining imipenem/cilastatin with relebactam, for treating complicated urinary tract and intra-abdominal infections.
  • Health authorities emphasize Recarbrio should be reserved as a last-resort treatment option, specifically for cases where limited or no alternative antibacterial drugs are available.
  • The drug targets resistant gram-negative bacteria including Pseudomonas and Klebsiella species, with relebactam designed to overcome carbapenem resistance mechanisms.

Medidata CEO Outlines Vision for Clinical Trial Evolution: Balancing Innovation with Regulatory Compliance

  • Medidata's leadership emphasizes the need for measured innovation in clinical trials, highlighting the delicate balance between rapid advancement and patient safety in pharmaceutical development.
  • The company is actively engaging with regulatory bodies like FDA and EMA to implement patient-centric approaches, including e-consent solutions and synthetic control arms to improve trial efficiency.
  • Industry transformation is being driven by regulatory demands for comprehensive data, payer focus on value-based outcomes, and the emergence of potentially curative compounds requiring nimbler development processes.

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